The Mouse Has Landed

I will literally do anything to help Maxwell even if it requires smuggling mice internationally.

Before I even dive into this blog post, BuzzFeed has done an incredible job telling my story – from developing a gene therapy to creating mutant mice in China – to help cure SLC6A1. If anyone would like to help, please share my GoFundMe on social media (FB, Twitter, LinkedIn) –every share raises awareness and helps to fundraise the massive amount required to advance a clinical trial ($4,000,000 – $7,000,000). The bottom line is the more money we raise, the faster we can go and the more kids we can treat.

Maxwell was diagnosed with a rare neurological disease known as SLC6A1 last summer. I spent my career in equity analysis and I knew nothing about science. My last scientific experiment involved sea monkeys in the 4th grade. Although mice with SLC6A1 existed, scientists told me early on that I needed a greater variety of genetically engineered mice to test the treatment. What does this mean in non-scientist speak?

We needed more mice that were representative of patients with the disease. A mouse can cost up to $50,000in the United States and quite simply, we did not have the money to invest in a mouse. We had just received the news that we needed to raise $4,000,000 – $7,000,000 to advance a gene replacement therapy. Buying a rodent that equated to the cost of Range Rover was not realistic.

I contacted some brilliant scientists in China interested in SLC6A1 and explained my situation. A very compassionate scientist agreed to make Maxwell’s mouse totally free of charge. The process of making a mouse can be convoluted and time consuming. A human mutation is implanted into the mouse DNA using a technique called CRISPR. These mice can then be used as pre-clinical models for developing and validating novel therapies. It also dramatically reduces the burden on pediatric patients to undergo invasive medical testing. These mice save innocent lives.

The mouse was finished in September and the Chinese team informed me of the good news. Even better, the mouse closely mirrored Maxwell’s symptoms (sometimes it doesn’t happen).

But there was one giant problem, the mouse lived in Shanghai and we needed to get it to the United States.

There was a lot of red tape surrounding the transfer process. I am an impatient person by nature, but have virtually no patience when it comes to slowing down the process to help Maxwell.

After some deliberation, I decided to get the mouse in China myself. After all, the TSA has never caught my hair spray. This is my son and the risk/reward is well worth it in my opinion.

Luckily, I didn’t have to go to China. Scientists from Vanderbilt stepped into save the day. Vanderbilt boasts the top animal facilities in the nation and was able to coordinate a quick transfer of the mouse. The airfare was $3,000 so let’s hope it received an extra-large cheese platter, warm towel and suitable movie options.

The mice flew via China Eastern Airlines directly from Shanghai to New York City. My household nervously tracked the flight all day. The flight was delayed by an hour and I refreshed my phone at least 100 times. Mark and I opened champagne when the flight safely landed. After a short stay in quarantine, the mouse will begin breeding and advance our research efforts as we battle to cure SLC6A1 forever.

It is astounding to think of the ways the world has come together to help cure SLC6A1.I could thank the group of Chinese scientists every day for the rest of my life and it would still not be an adequate display of my appreciation.

I have said this many times, but I have seen the best of humanity every day as a result of Maxwell’s diagnosis.

JP Morgan hosts the world’s largest healthcare conference every year in San Francisco. If you want to learn about the latest innovation and advances in biotech, the conference is the place to be. Not only Read more…